Clinical Study of CLL-1 CAR-T in the Treatment of Children With R/R AML

A Single-Center Clinical Study Evaluating the Safety and Preliminary Efficacy of CLL-1 CAR-T in the Treatment of Children With Relapsed/Refractory Acute Myeloid Leukemia

A study to evaluate the safety and preliminary efficacy of CLL-1-targeted CAR-T cell therapy in children aged 3 to 18 years with relapsed or refractory acute myeloid leukemia (r/r AML).

Study Overview

• Status: Not yet recruiting
• Conditions: CAR-T; AML (Acute Myeloid Leukemia)
• Intervention / Treatment: Biological: CAR-T

Detailed Description

It's a single-center, single-arm, investigator-initiated clinical study. After thorough consideration and signing of the informed consent form by the subject and/or their legal guardian(s), subjects meeting the eligibility criteria during the screening period will provide blood samples for CAR-T manufacturing. Following lymphodepleting chemotherapy, CAR-T cell infusion will be administered on D0.

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Yunyan He, PhD; Phone Number: +86 13607868275; Email: yunyanhe@aliyun.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Voluntarily sign the ICF and are expected to complete the study's follow-up examinations and procedures.
2. Aged 3 to 18 years (inclusive), and body weight ≥10 kg.
3. Diagnosis of AML according to the 2016 WHO classification, meeting the diagnostic criteria for relapse and refractoriness as per the "Chinese Guidelines for Diagnosis and Treatment of Relapsed/Refractory Acute Myeloid Leukemia (2017 Edition)", and currently having no clinically relevant treatment options or suitable registered clinical trials available.
4. Confirmation of CLL-1 expression ≥50% on AML blasts by flow cytometry.
5. Recovery from toxicities of prior therapies.
6. Karnofsky score (for age ≥16 years) ≥70 or Lansky score (for age <16 years) ≥50 at screening, and an expected survival >3 months.
7. Suitable function of the liver, kidneys, hematological system, lungs and heart is required.
8. Females subjects of childbearing potential must have a negative blood pregnancy test at screening and agree to use effective contraception during the study and within 1 year after the last dose of study drug.
9. Male subjects with reproductive potential must agree to use barrier contraception or practice complete abstinence until 1 year after the last study treatment.
10. Eligible blood cell specimen can be provided.

Exclusion Criteria:

1. Diagnosis of APL.
2. History of other malignancies within 3 years prior to screening, except for adequately treated carcinoma in situ of the cervix, papillary thyroid carcinoma, basal cell or squamous cell skin cancer, localized prostate cancer treated with radical surgery, and ductal carcinoma in situ treated with radical surgery.
3. Evidence of CNS involvement or cranial nerve pathology.
4. Subjects with active infections such as hepatitis B, hepatitis C, etc., are to be excluded.
5. Subjects with a history of severe allergies or known allergy to any drug component included in this study are to be excluded.
6. Subjects with severe cardiac diseases, refractory hypertension, active neurological autoimmune or inflammatory diseases, clinically significant active cerebrovascular disease, oncologic emergencies requiring urgent intervention, acute or chronic GVHD, or any uncontrolled infections requiring antibiotic therapy, etc., are unsuitable for enrollment.
7. Previous organ transplant or planned organ transplant (except for hematopoietic stem cell transplantation).
8. Received allo-HSCT within 6 weeks prior to screening.
9. Subjects who have recently undergone major surgery or plan to undergo major surgery, excluding diagnostic procedures and biopsies.
10. Subjects with severe mental disorders, alcoholism, or drug abuse.
11. Subjects who, in the judgment of the investigator, have other conditions that make them unsuitable for enrollment.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CAR-T; CAR-T therapy	Biological: CAR-T; A infusion of CLL-1 CAR-T cells following a lymphodepleting chemotherapy regimen.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Treatment-Related Adverse Events	Proportion of subjects experiencing all kinds of treatment-related AEs.	Up to 2 years.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Response Rate	Proportion of subjects achieving CR, CRi, morphologic leukemia-free state (MLFS), or PR.	Up to 2 years.
Duration of Response	Time from first assessment of CR, CRi, MLFS, or PR to first assessment of disease recurrence/progression or death from any cause.	Up to 2 years.
Relapse-Free Survival	For subjects achieving CR, CRi, or CR MRD-only. Time from achievement of response to relapse or death (any cause).	Up to 2 years.
Event-Free Survival	For all subjects. Time from cell infusion to treatment failure, relapse, or death (any cause).	Up to 2 years.
Overall Survival	For all subjects. Time from cell infusion to death from any cause.	Up to 2 years.
Proportion of CLL-1-Positive Tumor Cells	Changes in the proportion of CLL-1-positive tumor cells after CLL-1 CAR-T infusion.	Up to 2 years.

Collaborators and Investigators

• Sponsor: First Affiliated Hospital of Guangxi Medical University
• Collaborators: Guangzhou Bio-gene Technology Co., Ltd
• Investigators: Principal Investigator: Yunyan He, PhD, First Affiliated Hospital of Guangxi Medical University

Study record dates

Study Major Dates

• Study Start (Estimated): January 5, 2026
• Primary Completion (Estimated): October 31, 2028
• Study Completion (Estimated): October 31, 2028

Study Registration Dates

• First Submitted: December 22, 2025
• First Submitted That Met QC Criteria: January 5, 2026
• First Posted (Actual): January 13, 2026

Study Record Updates

• Last Update Posted (Actual): January 13, 2026
• Last Update Submitted That Met QC Criteria: January 5, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: CAR-T; R/R AML
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia, Myeloid; Leukemia; Hemic and Lymphatic Diseases; Leukemia, Myeloid, Acute
• Other Study ID Numbers: BG-CT-25-012

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No